Nucleic acids and procedures for the treatment of Pompe disease
- 专利权人:
- Synthena AG
- 发明人:
- GARCIA, Luis,AVRIL, Aurélie
- 申请号:
- ES14761668
- 公开号:
- ES2739850T3
- 申请日:
- 2014.09.10
- 申请国别(地区):
- ES
- 年份:
- 2020
- 代理人:
- 摘要:
- The invention relates to nucleic acids and methods for restoring acid alpha-glucosidase (GAA) activity in patients with Pompe disease using splice-switching technology.
- 来源网站:
- 中国工程科技知识中心